Real-world evidence enhances decision making

DISCLOSURES: No funding supported the writing of this commentary. The author has nothing to disclose.

high-quality comparative RWD effectiveness studies that were included in therapeutic class reviews.
We also interviewed managed care pharmacists and physicians and learned that there was keen interest in RWE for other decision processes besides the P&T process, especially those concerning safety and management of complex conditions. 3 At that time, the momentum for RWE was building and as apparent by the number of RWE studies published in JMCP. Over the course of 2019, an average of 6 (range: [3][4][5][6][7][8][9][10][11][12][13]  confirming findings from randomized controlled trials (RCTs), 7 evaluating subpopulations not well studied in The widespread availability of administrative claims and, now electronic medical records has enabled the ability to use this information to provide further insights into what and how care is being delivered and its associated cost. Before engaging in a discussion of the benefits of real-world evidence (RWE), it is necessary to state that not all real-world data (RWD) are RWE. In a seminal paper, Sherman et al. (2016) from the U.S. Food and Drug Administration (FDA) delineated between RWD and RWE. RWD is the collection of health-related information, and when stored electronically, these data can be used for analyses to answer a wide variety of research questions. 1 On the other hand, RWE is the transformation of RWD into evidence that can aid health care decision making at the patient, provider, and payer levels.
In 2017, my colleagues and I (Hurwitz et al.) published a study in JMCP that evaluated pharmacy and

Editor's note
The year 2020 marks the 25th anniversary of the Journal of Managed Care + Specialty Pharmacy. To commemorate this milestone, we are publishing a series of articles that document the changes to the journal and profession over the past 2 and a half decades. Each month we reprint an original article from a previous year, dating back to 1995. The reprinted articles feature topics of significance in our industry. Each reprinted article is accompanied by a contemporary reflection that will consider the historical significance of the topic, as well as the current and future state. One of the most important outcomes for health plans and payers are the results related to costs and economic outcomes. Approval of coverage for technologies are overlaid with concerns that the product will fail to provide meaningful health benefits and will be a waste of resources.
Thus, "cost" is always an important outcome for health plans often not studied in RCTs. 3 15 Another issue to consider is whether data are the best evidence to answer a study question. While RWE studies can evaluate a wide spectrum of questions, not all questions can or should be answered with administrative claims or other electronic data. 16 Most problematic with observational studies of therapeutics is the issue of confounding by indication. Confounding occurs when the indication is related to both the outcome and treatment. Because prescribed medicine use is driven by indication, comparisons between therapies can be driven by differences in disease severity, not necessarily due the treatments. In addition, administrative claims data do not contain detailed clinical data, and while the implementation of International Classification of Diseases, Tenth Revision has greatly improved precision, data are subject to upcoding, undercoding, and errors in coding. A number of other potential biases with observational data must be considered as well, including selection bias, maturation, and regression to the mean.
Despite the limitations of RWE, the benefits can be substantial. For example, clinical trials suffer from low generalizability. Payers and decision makers want to know if there are subgroups or special populations where the intervention is effective, and perhaps more importantly, when it isn't. Clinical trials are often of short duration and have restrictive inclusion/exclusion criteria. Findings from these studies are often limited to a subset of the population who will receive a therapy, leaving many gaps in evidence. RWE can help fill the gaps. Because of its relatively low cost and short time to conduct, RWE can provide additional evidence on the effectiveness and safety of medical innovations.
While RWE is not a panacea for all evidence gaps and decision-maker needs, it can provide timely information in some situations. That stated, not every question can be answered with RWD. Data must be fit for the purpose. No amount of manipulation or analysis can make data better than they are. The key is clearly defining the research question before starting the analysis.
In summary, while RWE is not always the best source of evidence (especially for determining efficacy), widespread availability of health care encounters and outcomes via electronic resources is leading to an explosion of studies to support decision making and managed care pharmacy. As the tools to analyze data continue to improve, evidence will be available to inform our decisions, perhaps even in real time. While RCTs will continue to serve as the principal source for initial review and coverage decisions, RWE will be useful in supplementing and enhancing our understanding of the effect of various technologies and interventions.